Background. Impairment of the endocrine and exocrine function of the pancreas is a common complication in thalassemia major (TM). Multiecho T2* Magnetic Resonance Imaging (MRI) allows the reproducible and noninvasive assessement of pancreatic iron overload. However, there are no prospective studies describing the changes of pancreatic T2* values. So, our aim was to describe the changes in pancreatic T2* values over a follow-up (FU) of 18 months and to evaluate prospectively the effectiveness of the three iron chelators in monotherapy.

Methods. We selected 22 TM patients consecutively enrolled in the Myocardial Iron Overload in Thalassemia (MIOT) Network who had received only one chelator in monotherapy between the two MRI scans. Pancreatic iron burden was measured using a T2* gradient-echo multiecho sequence. The images were analyzed using a previously validated, custom-written software (HIPPO-MIOT®).

Results. Three groups of patients were identified: 9 patients (5 females, mean age 32.8±8.6 years) treated with desferioxamine (DFO – mean dosage 44.8±3.8 mg/kg/die), 6 patients (2 females, mean age 36.3±6.5 years) treated with deferiprone (DFP– dosage 75mg/kg/die) and 7 patients (6 females, mean age 30.4±9.1 years) treated with deferasirox (DFX – mean dosage 28.2±4.6 mg/kg/die). The percentage of patients with a excellent/good compliance was comparable among the groups.

All patients under DFO therapy showed at the baseline MRI pancreatic iron overload (T2*<26 ms) and at the FU only one had a normal pancreas T2* value (Figure 1, left). Globally there was a significant increment in the pancreatic T2* values (mean difference: 3.66±3.94; P=0.021).

In the DFP group at baseline 5 patients showed pancreatic iron and none recovered at the follow up (Figure 1, center). The patient with a normal baseline pancreatic T2* value, maintained it at the FU. For the subgroup with pancreatic iron at the baseline, there was a significant increment in the pancreatic T2* values (mean difference: 3.99±2.05; P=0.043).

In the DFX group 5 patients showed at the baseline pancreatic iron and although the pancreatic T2* increased for all of them, the normal value was not reached at the follow up (Figure 1, right). Both the patients who showed no pancreatic iron overload at the baseline maintained at the FU the same status. For the subgroup with pancreatic iron at the baseline, there was a significant increment in the pancreatic T2* values (mean difference: 2.48±3.06; P=0.043).

Conclusion: Prospectively in TM patients at the dosages used in the clinical practice all three chelators in monotherapy allowed a significant reduction in pancreatic iron. Further prospective studies involving more patients are needed to establish which is the most effective drug.

Disclosures

Pepe:Novartis: Speakers Bureau; ApoPharma Inc.: Speakers Bureau; Chiesi: Speakers Bureau.

Author notes

*

Asterisk with author names denotes non-ASH members.

Sign in via your Institution